A $7 million grant from the National Institutes of Health (NIH) will help researchers at Nationwide Children's Hospital translate new scientific findings and technological developments into novel treatments for the muscular dystrophies. The grant designates Nationwide Children's Hospital as a Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, one of three national award recipients in 2010.
The award explores and refines therapeutic strategies that have shown promise in animal models of muscular dystrophy. Studies include further research in the laboratory as well as clinical trials to assess efficacy in patients. These efforts include replacing defective genes responsible for muscular dystrophy with healthy ones. Other trials use substitute or surrogate genes to restore muscle function. A third approach attempts to repair gene function using specially designed medications. All the studies aim to slow the progression of muscular dystrophy.
In 2001, the United States MD-CARE Act specified a number of provisions for expanding and intensifying research on muscular dystrophy, notably the NIH's establishment of centers of excellence for research on these muscle diseases. The Muscular Dystrophy Cooperative Research Centers (MDCRCs) program was subsequently developed in honor of Senator Paul D. Wellstone, a champion of muscular dystrophy research. MDCRCs promote basic, translational and clinical research and provide important resources that can be shared within the national muscle biology and neuromuscular research communities. The MDCRCs also serve as focal points for research collaborations and provide training and guidance for basic and clinical researchers.
The MDCRC will allow Nationwide Children's researchers to further develop methods to overcome immune barriers to gene correction for Duchenne muscular dystrophy (DMD). Jerry Mendell, MD, director, Center for Gene Therapy and Chris Walker, PhD, director, Center for V
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Nationwide Children's Hospital